Scientists at Florida International University have developed a promising new compound that could slow the progression of Parkinson’s disease by targeting a key process inside cells.
The research centres on dynamin-related protein 1 (DRP1), which helps control how mitochondria – often described as the powerhouses of cells – divide and function. When DRP1 becomes overactive, it can lead to impaired mitochondrial function, inflammation and cell damage linked to disease.
In preclinical tests the compound, CTS2444-32, reduced DRP1 activity by up to 45%, helping limit damage in brain disease models.
Kim Tieu, a professor of environmental health sciences at FIU Robert Stempel College of Public Health & Social Work, has spent years studying DRP1’s role in Parkinson’s, a disease marked by tremors, stiffness and the loss of brain cells.
“DRP1 plays an important role in mitochondrial division,” Tieu said. “But when it gets too active, it causes the mitochondria to split more than they should, which can lead to mitochondrial dysfunction, inflammation and cell death – key factors in how Parkinson’s disease progresses.”
Reduced inflammation
In Parkinson’s models, CTS2444-32 reduced inflammation in the brain and lowered levels of toxic proteins such as alpha-synuclein, which are closely linked to the disease.
“Unlike current treatments that simply replace the missing chemical to relieve symptoms, this compound has the potential to actually protect brain cells and slow down how Parkinson’s disease progresses,” Tieu said.
The compound was identified by screening a library of 35 million compounds. Researchers said the compound could have even broader potential.
The team has secured a patent and is now working to refine the compound so it can be safely used in humans. Additional studies are underway, including testing in larger animals to meet FDA requirements. Clinical trials could begin within a year.
If successful, the treatment could improve outcomes for patients. People with Parkinson’s may see slower disease progression.
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